This story was produced by Postmedia's advertising department on behalf of the Canadian Pulmonary Fibrosis Foundation for commercial purposes. Postmedia's editorial departments had no involvement in the creation of this content.

Canada needs a national strategy for rare diseases

Getting access to, and funding for, treatments for rare diseases has been a long-standing challenge in Canada. Unlike more common diseases, where economies of scale allow for easier access to research and funding support, rare-disease groups have been facing an uphill battle that spans years.

Rare diseases are classified as those that affect fewer than one in 2,000 people. Overall, according to the Canadian Organization for Rare Disorders (CORD), there are close to 7,000 rare diseases.

Canada stands apart as a country that does not yet have a policy in place to help bring treatments for rare diseases. The United States has had its Orphan Drug Policy since 1983. Japan (1993), Australia (1997), and Europe (2000) have been equally forthcoming in developing frameworks to support rare disease research, drug development and access to treatment.

According to CORD, of the 307 orphan drugs currently approved in the United States and Europe only 60% make their way into Canada; and most get approved up to six years later than in the United States and Europe. As a result, people with rare disorders are missing out on treatment that could save or significantly improve their lives, says Durhane Wong-Rieger, CORD president and chief executive officer in Toronto.

This is an issue that was brought to light in the 1990s, she adds. “Up until the mid-1980s nobody was making drugs for rare diseases because it was too difficult to find patients, and too expensive. The United States recognized that the need to give special assistance to researchers and drug companies was important enough to establish a policy.”

Since 2006, rare-disease organizations have joined forces to help devise a Canadian Orphan Drug Regulatory Framework policy. Wong-Rieger anticipates that Health Canada will approve the policy will pass this fall, barring further delays.

Even when a drug is approved, patients are in no position to pay for them, she says. “These drugs could cost anywhere from $20,000 to $100,000 a year or more. You would either have to have a good private insurance plan or a public health system to pay for it. That’s a problem for us in Canada.”

While the framework will play its part in helping to fast-track approvals, funding will remain an issue. “Even if 80% of drugs coming to Canada get approved, provinces may say no to funding them.”

One significant stumbling block has been the measurement criteria applied for funding approval, she explains. “The calculations are quite complex. Because rare diseases are very expensive compared to more common drugs, they can’t meet the current cost-effectiveness threshold used to determine approval.”

The answer, she believes, is a “special pathway” for approvals, in which drugs will be evaluated based on benefits to patients versus a simple cost-per-patient basis.

Wong-Rieger counters the traditional methodology with the fact that rare-disease treatments are extremely targeted and only prescribed to patients who respond to them. This is not the case for more common treatments. “There is a great deal of money wasted today where the system is prescribing drugs for people where there is no benefit. Rare-diseases treatments are only prescribed where there is a benefit.”

Considering in detail who might benefit and monitoring responses will, in fact, save considerable costs in terms of hospital care. “Some of the care requirements in hospitals for these patients is extremely expensive,” she says.

Measuring value on a benefits basis is already a practice in other areas of health care. For example, cardiac surgery has reduced deaths from heart disease by 50%. “No one says that the surgery is too expensive because the benefit is clear,” Wong-Rieger says. “Treatments for rare diseases should be considered in the same light.”

Diseases such as cystic fibrosis and muscular dystrophy have been among the more highly publicized afflictions involved in an uphill battle for early treatment coverage, she adds.

“The sad part of all this is that we are holding these patients hostage while negotiations carry on. Treatments are not meant to be weapons in this battle.”

This story was produced by Postmedia’s advertising department on behalf of the Canadian Pulmonary Fibrosis Foundation for commercial purposes. Postmedia’s editorial departments had no involvement in the creation of this content.